A Pilot Study of HP802-247 in Dystrophic Epidermolysis Bullosa

NCT ID: NCT01528306

Last Updated: 2012-05-16

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-03-31
• Study Completion Date: 2013-07-31

Brief Summary

This is a pilot study designed to see if HP802-247, an investigational treatment with living human skin cells, helps to heal blisters or wounds in subjects with Dystrophic Epidermolysis Bullosa (DEB). The durability of the skin in healed wounds treated with HP802-247 will also be assessed.

Conditions

Dystrophic Epidermolysis Bullosa

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

HP802-247

Group Type: EXPERIMENTAL

HP802-247

Intervention Type: BIOLOGICAL

Wounds up to 12 cm2: 1 spray each component (0.5 x 106 cells per mL allogeneic human keratinocytes and fibroblasts) applied weekly as a topical spray for up to 4 weeks in each of 2 treatment periods

Larger wounds:

(>12 cm2 and ≤ 24 cm2) 2 sprays each; (>24 cm2 and ≤ 36 cm2) 3 sprays each; (>36 cm2 and ≤ 48 cm2) 4 sprays each

Placebo (Vehicle)

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Wounds up to 12 cm2: 1 spray each component (thrombin solution and fibrinogen solution) applied weekly as a topical spray for up to 4 weeks in each of 2 treatment periods

Larger wounds:

(>12 cm2 and ≤ 24 cm2) 2 sprays each; (>24 cm2 and ≤ 36 cm2) 3 sprays each; (>36 cm2 and ≤ 48 cm2) 4 sprays each

Interventions

HP802-247

Wounds up to 12 cm2: 1 spray each component (0.5 x 106 cells per mL allogeneic human keratinocytes and fibroblasts) applied weekly as a topical spray for up to 4 weeks in each of 2 treatment periods

Larger wounds:

(>12 cm2 and ≤ 24 cm2) 2 sprays each; (>24 cm2 and ≤ 36 cm2) 3 sprays each; (>36 cm2 and ≤ 48 cm2) 4 sprays each

Intervention Type: BIOLOGICAL

Placebo

Wounds up to 12 cm2: 1 spray each component (thrombin solution and fibrinogen solution) applied weekly as a topical spray for up to 4 weeks in each of 2 treatment periods

Larger wounds:

(>12 cm2 and ≤ 24 cm2) 2 sprays each; (>24 cm2 and ≤ 36 cm2) 3 sprays each; (>36 cm2 and ≤ 48 cm2) 4 sprays each

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• Written consent must be obtained from a parent or legally authorized representative. Assent will be obtained according to local requirements.
• Subjects with a clinical diagnosis of DEB with recurring blisters, ≥ 2 years of age at Screening. Subjects may be of either sex and of any race or skin type provided that their skin color, in the opinion of the Investigator, will not interfere with the study assessments.
• A parent or legally authorized representative must be willing and able to ensure subject is present for all required study visits.
• A parent or legally authorized representative must be able to follow instructions.
• Have an open wound for ≤ 72 hours that:

• is still open and has not scabbed or crusted over
• has no dermatologic disease and/or condition in the treatment area (other than DEB with recurring blisters), including active or recent infection (within 7 days) that may be exacerbated by treatment, require treatment with antibiotics / antifungals /antivirals, require a surgical intervention, or cause difficulty with examination
• is ≥ 4 cm² and ≤ 48 cm2 in total area (open portion)
• Females of childbearing potential (defined as post-menarcheal as documented in the medical history) may participate in the study if they meet all of the following conditions:

• they are not breast feeding;
• they have a negative urine pregnancy test at Week 1 Period 1;
• they agree to undertake scheduled urine pregnancy tests at Week 1 for Periods 1, 2, & 3 and at study exit
• they do not intend to become pregnant during the study;
• they are using adequate birth control methods and they agree to continue using those methods for the duration of the study
• A parent or legally authorized representative must be willing and able to ensure subject is present for all required study visits (for minor subjects)
• A parent or legally authorized representative must be able to follow instructions (for minor subjects)

Exclusion Criteria

• Contraindications or hypersensitivity to the use of the test article, their components (e.g., aprotinin, fibrinogen), or substances used in the manufacture of the test article (e.g., penicillin, streptomycin, amphotericin B, bovine serum albumin).
• Therapy with another investigational agent within thirty (30) days of the Screening Visit, or during the study.
• Have uncontrolled intercurrent or chronic illness that, in the opinion of the Investigator, would limit compliance with study requirements, represents a potential safety risk, or require treatment with an excluded drug/treatment.
• The Investigator or Medical Monitor may declare any subject ineligible for a valid medical reason.

• Minimum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Healthpoint

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Herbert B Slade, MD

Role: STUDY_CHAIR

Healthpoint

Jaime E Dickerson, PhD

Role: STUDY_DIRECTOR

Healthpoint

Herbert B Slade, MD

Role: PRINCIPAL_INVESTIGATOR

Healthpoint

Other Identifiers

802-247-09-026

Identifier Type: -

Identifier Source: org_study_id